Stem Cell Clinic

Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma – Curetoday.com

By Stem Cell Treatment

Several drugs that work by targeting genetic alterations in cancer cells have won recent approval from the Food and Drug Administration as treatments for patients with diffuse large B-cell lymphoma (DLBCL).

Dr. Germame Ajebo, assistant professor of medicine at Georgia Cancer Center at Augusta University, shared information on novel treatments for the disease during the recent virtual CURE Educated Patient Leukemia & Lymphoma Summit.

In his talk, Ajebo focused on drugs meant for use in disease that has recurred or become resistant to previous treatments.

DLBCL is a usually aggressive form of the blood cancer known as a B-cell non-Hodgkin lymphoma, which affects the immune system. The disease causes rapid growth of tumors in the lymph nodes, spleen, liver, bone marrow or other organs.

Approved by the FDA within the last two years to treat aggressive DLBCL are oral Xpovio (selinexor), Polivy (polatuzumab vedotin-piiq) and Monjuvi (tafasitamab-cxix). In addition, an immunotherapy, the chimeric antigen receptor (CAR)-T cell therapy Yescarta (axi-cel), was approved to treat the disease in 2017, Ajebo reported.

Read more: Monjuvi-Revlimid Combination Approval Fills Unmet Need for Certain Patients with DLBCL.

Xpovio is a nuclear export inhibitor, which prevents cancerous cells from pushing tumor-suppressing proteins out of their nuclei. This results in tumor suppressors accumulating in the nucleus, where they can work to kill the cell.

In the phase 2b clinical trial that led to its approval which administered Xpovio by itself to 134 previously treated older adult patients the partial response rate (including those with tumor shrinkage) was 16%, the complete response rate (including those with no sign of cancer remaining) was 13% and the rate of stable disease (including patients with no progression of cancer) was 8.2%, Ajebo reported. Looking at all patients who had partial or complete responses, 38% responded for at least six months and 15% for at least 12 months.

The most common side effects that were serious or worse were low blood counts, Ajebo summarized. Other serious side effects included nausea, vomiting, diarrhea, weight loss, dizziness and infections.

Polivy is an antibody-drug conjugate that uses a targeted drug to deliver a potent chemotherapy directly to cancer cells.

It was approved based on the results of a phase 2 study of 80 previously treated patients who were divided into equally sized groups to receive the chemotherapy Treanda (bendamustine) and the targeted drug Rituxan (rituximab) with or without Polivy every 21 days for six cycles. At the end of treatment, 40% of those receiving the triplet combination had experienced a complete response, compared with 18% of those receiving Treanda and Rituxan alone. In the 63% of patients who achieved a best overall response at any point in the study while receiving the drug triplet, 48% had a response that lasted at least 12 months and 64% responded for at least six months, Ajebo noted.

Major side effects, he said, included tingling or weakness in the extremities, low blood counts, liver toxicity and tumor lysis syndrome, a condition that can damage organs due to blood chemistry issues arising from the quick destruction of tumor cells. The most common side effects of any severity included low blood counts, fatigue, diarrhea and fever.

Monjuvi, a targeted drug that inhibits the activity of the DLBCL-fueling protein CD19, was approved based on results of the phase 2 L-MIND study that demonstrated a 43% complete response rate and an 18% partial response rate in 80 previously treated adult patients who were prescribed the drug along with the targeted medication Revlimid (lenalidomide), with a median duration of response of 21.7 months. The drug is approved in combination with Revlimid for patients with recurrent or resistant DLBCL who are not eligible for, or did not agree to undergo, bone marrow transplant using their own stem cells, Ajebo said.

Serious side effects occurred in 52% of patients, he said, and included low blood counts and infections. The drug caused fatal reactions in 5% of patients, including stroke, respiratory failure, progressive multifocal leukoencephalopathy (a virus that infects the brain) and sudden death.

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Novel Targeted Drugs are Changing the Treatment of Diffuse Large B-Cell Lymphoma - Curetoday.com

Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation at 62nd American…

By Stem Cell Treatment

November 05, 2020 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

BOSTONandLONDON, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced the presentation of new clinical data at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting to be held virtually December 5-8, 2020. The oral presentation will highlight data from the first patient treated in the ongoing proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).

MPS-IIIA is a progressive, life-threatening metabolic disease with no approved treatment options, said Professor Robert Wynn, chief investigator at The Royal Manchester Childrens Hospital, part of Manchester University NHS Foundation Trust. We are pleased to see encouraging initial results, including evidence of engraftment of gene-modified cells, an important first step in the investigation of whether OTL-201 could address critical unmet needs for patients with MPS-IIIA. We look forward to continuing to advance this program to add to the growing body of evidence supporting the use of HSC gene therapy to treat severe neurometabolic conditions.

Preliminary results from the first patient treated with OTL-201 show evidence of engraftment of gene-modified cells, supra-physiological N-sulphoglucosamine sulphohydrolase (SGSH) enzyme expression in multiple lineages, and reduction of heparan sulfate in plasma, cerebrospinal fluid and urine over an initial three-month follow-up period. Additional follow-up data and an update on the trial status will be shared at the time of the oral presentation.

Oral Presentation Details:

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress NCT04201405 Publication number: 676 Session: 801. Gene Editing, Therapy and Transfer I Date and time: Monday, December 7, 2020; 12:45 p.m. PT

Abstracts are available online at the ASH Annual Meeting website.

About OTL-201 and MPS-IIIA Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides, including heparan sulfate. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.1 There are currently no approved treatment options for MPS-IIIA. OTL-201 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patients cells.

About Orchard Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

About Manchester University NHS Foundation Trust Manchester University NHS Foundation Trust is one of the largest NHS trusts in England and a leading provider of specialist healthcare services. Its nine hospitals are home to hundreds of world class clinicians and academic staff committed to finding patients the best care and treatments. More information is available at http://www.mft.nhs.uk.

Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

1Lavery, C., Hendriksz, C.J. & Jones, S.A. Mortality in patients with Sanfilippo syndrome. Orphanet J Rare Dis 12, 168 (2017) doi:10.1186/s13023-017-0717-y

Excerpt from:
Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation at 62nd American...

bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -…

By Stem Cell Treatment

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Updated results from patients in Group C of the companys Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.

bluebird bio will also present updated long-term efficacy and safety results from the LTF-303 follow-up study; outcomes across genotypes; and outcomes in pediatric patients from Phase 3 studies HGB-207 and HGB-212 of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia) in TDT.

Data from across the companys multiple myeloma program will be presented. Presentations will include updated safety and efficacy results from the Phase 1 CRB-401 clinical study of idecabtagene vicleucel (ide-cel, bb2121) and preliminary data from the ongoing Phase 1 CRB-402 clinical study of bb21217, as well as subgroup analyses of the pivotal Phase 2 KarMMa study of ide-cel. Ide-cel and bb21217 are investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immune therapies being studied, in partnership with Bristol-Myers Squibb, for the treatment of adult patients with MM.

Sickle Cell Disease Data at ASH

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy Presenting Author: Julie Kanter, MD, University of Alabama at Birmingham, Birmingham, AL Date/Time: Oral #365, Sunday, December 6, 2020, 9:45 am PST

Resolution of Serious Vaso-occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy Presenting Author: Alexis A. Thompson, MD, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital, Chicago, IL Date/Time: Oral #677, Monday, December 7, 2020, 1:30 pm PST

The GRNDaD Registry: Contemporary Natural History data and an analysis of real-world patterns of use and limitations of Disease Modifying Therapy in adults with SCD Presenting Author: Alexandra Boye-Doe, MD, University of North Carolina School of Medicine, Chapel Hill, NC Date/Time: Poster #1730, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Transfusion-Dependent -Thalassemia Data at ASH

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent -Thalassemia: Results in Patients with up to 6 Years of Follow-up Presenting Author: Janet L. Kwiatkowski, MD, MSCE, Director, Thalassemia Center at Children's Hospital of Philadelphia, Philadelphia, PA Date/Time: Oral #153, Saturday, December 5, 2020, 12:00 pm PST

Favorable Outcomes in Pediatric Patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) Studies of betibeglogene autotemcel Gene Therapy for the Treatment of Transfusion-dependent -thalassemia Presenting Author: Alexis A. Thompson, MD, MPH, Hematology Section Head, Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago, IL Date/Time: Oral #154, Saturday, December 5, 2020, 12:15 pm PST

Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent -Thalassemia in the Phase 3 HGB-207 Study Presenting Author: John B. Porter, MA, MD, FRCP, FRCPath, Head of Red Cell Unit, University College London Hospital, London, UK Date/Time: Poster #776, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Response of patients with transfusion-dependent -thalassemia (TDT) to betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy based on HBB genotype and disease genetic modifiers Presenting Author: Mark C. Walters MD, Medical Director, Jordan Family Center for BMT & Cellular Therapies Research, UCSF Benioff Childrens Hospital Oakland, Oakland, CA Date/Time: Poster #1699, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Multiple Myeloma Data at ASH

Updated results from the Phase I CRB-402 study of anti-BCMA CAR-T cell therapy bb21217 in patients with relapsed and refractory myeloma: correlation of expansion and duration of response with T cell phenotypes Presenting Author: Melissa Alsina, MD, Department of Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL Date/Time: Oral #130, Saturday, December 5, 2020, 9:45 am PST

Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study Presenting Author: Yi Lin, MD, PhD, Division of Hematology, Mayo Clinic, Rochester, MN Date/Time: Oral #131, Saturday, December 5, 2020, 10:00 am PST

Secondary Quality-of-Life Domains in Patients With Relapsed and Refractory Multiple Myeloma Treated With the BCMA-Directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel; bb2121): Results from the KarMMa Clinical Trial Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA Date/Time: Oral #437, Sunday, December 6, 2020, 12:15 pm PST

Efficacy and Safety of Idecabtagene Vicleucel (ide-cel, bb2121) in Elderly Patients with Relapsed/Refractory Multiple Myeloma: KarMMa Subgroup Analysis Presenting Author: Jess Berdeja, MD, Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN Date/Time: Poster #1367, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Characterization of Cytokine Release Syndrome in the KarMMa Study of Idecabtagene Vicleucel (ide-cel, bb2121) For Relapsed and Refractory Multiple Myeloma Presenting Author: Ankit Kansagra, MD, Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, Dallas, TX Date/Time: Poster #1378, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Molecular and Phenotypic Profiling of Drug Product and Post-infusion Samples from CRB-402, an Ongoing: Phase I Clinical Study of bb21217 a BCMA-directed CAR T Cell Therapy Presenting Author: Olivia Finney, PhD, Associate Director, Immunotherapy, bluebird bio Date/Time: Poster #1401, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Effects of Prior Alkylating Therapies on Preinfusion Patient Characteristics and Starting Material for CAR T Cell Product Manufacturing in Late-Line Multiple Myeloma Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb, Princeton, NJ Date/Time: Poster #1405, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

KarMMa-4: Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy, in High-Risk Newly Diagnosed Multiple Myeloma Presenting Author: Saad Z. Usmani, MD, Director, Clinical Research in Hematologic Malignancies, Levine Cancer Institute/Atrium Health, Charlotte, NC Date/Time: Poster #1418, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Healthcare Resource Utilization and Cost of Cytokine Release Syndrome and Neurologic Events in Patients with Relapsed and Refractory Multiple Myeloma Receiving the BCMA-directed CAR T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121) in the KarMMa Trial Presenting Author: Parmeswaran Hari, MD, Medical College of Wisconsin, Milwaukee, WI Date/Time: Poster #1598, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

A Matching-Adjusted Indirect Comparison of Efficacy Outcomes for Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-directed CAR T Cell Therapy Versus Conventional Care in Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Presenting Author: Nina Shah, MD, University of California San Francisco, San Francisco, CA Date/Time: Poster #1653, Saturday, December 5, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) Responses Are Characterized by Early and Temporally Consistent Activation and Expansion of CAR T Cells With a T Effector Phenotype Presenting Author: Nathan Martin, PhD, Bristol Myers Squibb, Princeton, NJ Date/Time: Poster #2315, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

KarMMa-3: A Phase 3 Study of Idecabtagene Vicleucel (ide-cel, bb2121), a BCMA-Targeted CAR T Cell Therapy Versus Standard Regimens in Relapsed and Refractory Multiple Myeloma Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium Date/Time: Poster #2323, Sunday, December 6, 2020, 7:00 am 3:30 pm PST

Idecabtagene Vicleucel (ide-cel, bb2121) in Relapsed and Refractory Multiple Myeloma: Analyses of High-Risk Subgroups in the KarMMa Study Presenting Author: Noopur S. Raje, MD, Massachusetts General Hospital, Boston, MA Date/Time: Poster #3234, Monday, December 7, 2020, 7:00 am 3:00 pm PST

Health State Utility Valuation in Patients with Triple-Class Exposed Relapsed and Refractory Multiple Myeloma Treated with the BCMAdirected CAR T Cell Therapy, Idecabtagene Vicleucel (idecel, bb2121): Results from the KarMMa Trial Presenting Author: Michel Delforge, MD, PhD, University Hospital Leuven, Leuven, Belgium Date/Time: Poster #3465, Monday, December 7, 2020, 7:00 am 3:00pm PST

Abstracts outlining bluebird bios accepted data at ASH are available on the ASH conference website.

About LentiGlobin for SCD (bb1111)

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

LentiGlobin for SCD was designed to add functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once patients have the A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbA-T87Q) that decreases the proportion of HbS, with the goal of reducing sickled red blood cells, hemolysis and other complications.

As of March 3, 2020, a total of 37 patients have been treated with LentiGlobin for SCD to-date in the HGB-205 (n=3) and HGB-206 (n=34) clinical studies. The HGB-206 total includes: Group A (n=7), B (n=2) and C (n=25).

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

The U.S. Food and Drug Administration (FDA) granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD. LentiGlobin for SCD continues to be evaluated in the ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About betibeglogene autotemcel

Transfusion dependent beta-thalassemia (TDT) is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT require chronic blood transfusions to maintain adequate Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel (beti-cel) adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbA-T87Q, which is gene therapy-derived adult hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

As of March 3, 2020, a total of 60 pediatric, adolescent and adult patients, including 11 patients with at least 5 years of follow-up, across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. In studies of beti-cel, patients were assessed for transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. FDA granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the United States. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.

About idecabtagene vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio. Ide-cel was granted accelerated assessment by the European Medicines Agency (EMA) on March 26, 2020, and the Marketing Authorization Application (MAA) was validated by the EMA on May 20, 2020. The FDA accepted the ide-cel Biologics License Application (BLA) for priority review on September 22, 2020.

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with RRMM in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

CRB-401 (NCT02658929) is an open-label Phase 1 study evaluating the preliminary safety and efficacy of ide-cel in patients with relapsed and refractory multiple myeloma (RRMM). The primary endpoint of the study is safety. CRB-401 was designed as a two-part (dose escalation and dose expansion) study to determine the maximum tolerated dose and further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose; these findings established the recommended dose of the Phase 2 KarMMa trial. All patients have been treated in the study and follow-up is ongoing.

In addition to the pivotal KarMMa and CRB-401 trials, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) exploring ide-cel combinations and activity in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is not approved for any indication in any geography.

About bb21217

bb21217 is an investigational BCMA-targeted CAR T cell therapy that uses the ide-cel CAR molecule and is cultured with the PI3 kinase inhibitor (bb007) to enrich for T cells displaying a memory-like phenotype with the intention to increase the in vivo persistence of CAR T cells. bb21217 is being studied for patients with multiple myeloma in partnership with Bristol Myers Squibb.

bluebird bios clinical development program for bb21217 includes the ongoing Phase 1 CRB-402 study. CRB-402 is the first-in-human study of bb21217 in patients with relapsed and refractory multiple myeloma (RRMM), designed to assess safety, pharmacokinetics, efficacy and duration of effect. CRB-402 is a two-part (dose escalation and dose expansion), open-label, multi-site Phase 1 study of bb21217 in adults with RRMM. For more information visit: clinicaltrials.gov using identifier NCT03274219.

bb21217 is not approved for any indication in any geography.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent -thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD, and the potential for idecabtagene vicleucel and bb21217 to treat multiple myeloma; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

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bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition -...

Regenerative Medicine Stem Cells Market Forecast Revised in a New FMI Report as COVID-19 Projected to Hold a Massive Impact on Sales in 2019 – KYT24

By Stem Cell Medicine

This report analyzes the current and future prospects of the regenerative medicine across the globe. Regenerative medicine is considered as an emerging field of medical science that aims to regenerate, repair or replace damaged tissue and organs.

The research is a robust combination of primary and secondary research. Primary research formed the bulk of our research efforts with information collected from telephonic interviews and interactions via e-mails. Secondary research involved study of company websites, annual reports, press releases, stock analysis presentations, and various international and national databases. The report provides market size in terms of US$ Mn for each segment for the period from 2015 to 2019, considering the macro and micro environmental factors. Growth rates for each segment within the global regenerative medicine market have been determined after a thorough analysis of past trends, demographics, future trends, technological developments, and regulatory requirements.

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The market report comprises an elaborate executive summary, which includes a market snapshot that provides information about various segments and sub-segments of the market. It also provides information and data analysis of the global regenerative medicine market with respect to market segments based on the technology type, application type and geographic analysis.This report includes estimation of theglobal Regenerative Medicine Stem Cells marketin terms of value (USD million) for the period 2015 to 2019. In addition, current trends and recent developments are taken into consideration while determining the growth rate of the global Regenerative Medicine Stem Cells market. The report provides market size and forecast for each segment, sub-segment and geographic region with CAGR % for the period 2015 to 2019.

The global market for regenerative medicine is segmented based on technology as stem cell therapy, biomaterials and tissue engineering. In 2012, biomaterials segment accounted for the largest market share in the global Regenerative Medicine Stem Cells market owing to favorable reimbursement policies and strong demand of biomaterials in the global market. However, high cost associated with biomaterials is a factor that would restrict the global market demand to some extent during the study period.

In addition, based on applications the global market for Regenerative Medicine Stem Cells is segmented as bone graft substitute, osteoarticular diseases, allogeneic bones, autogenic bones and others. In 2012, bone graft substitute segment accounted for the largest market share in the global regenerative medicine bone and joint application market owing to growing demand of bone graft substitute in orthopaedic surgeries. However, post implantation rejection associated with bone graft substitute is considered as a crucial factor that would restrict the global market demand of bone graft substitute.

Geographically, the global Regenerative Medicine Stem Cells market has been segmented into four regions: North America, Europe, Asia-Pacific and Rest of the World (RoW). Market size, forecast and CAGR (%) for each region have been provided. A detailed qualitative analysis of the factors responsible for driving and restraining the growth of the Regenerative Medicine Stem Cells market and future opportunities are provided in the report. A PESTLE analysis has also been done while estimating individual geographies in order to provide current as well as future status. A list of recommendations for existing as well as new entrants has also been discussed in the study that is expected to help in decision-making.

Major market players profiled in this report includes Anika Therapeutics, Inc., Arthrex, Inc., Baxter International, Inc., CONMED Corporation, DePuy Synthes, Inc., Medtronic, Inc., Smith & Nephew PLC, Stryker Corporation and Zimmer Holdings, Inc.

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The global Regenerative Medicine Stem Cells market is segmented as follows:

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Regenerative Medicine Stem Cells Market Forecast Revised in a New FMI Report as COVID-19 Projected to Hold a Massive Impact on Sales in 2019 - KYT24

How the Election Could Affect Health Care and Drug Policy – Scientific American

By Stem Cell Medicine

With the winner of the presidency and party control of the Senate still unclear the morning after Election Day, the future of the nations health system remains uncertain. At stake is whether the federal government will play a stronger role in financing and setting the ground rules for health care coverage or cede more authority to states and the private sector.

Should President Donald Trump win and Republicans retain control of the Senate, Trump still may not be able to make sweeping changes through legislation as long as the House is still controlled by Democrats. But thanks to rules set up by the Senate GOP the ability to continue to stack the federal courts with conservative jurists who are likely to uphold Trumps expansive use of executive power could effectively remake the governments relationship with the health care system even without signed legislation.

The president has also pledged to continue hisefforts to get rid of the Affordable Care Act, and if the Supreme Court overturns the sweeping law as part of a challenge it will hear next week, the Republicans promise to protect people with preexisting medical conditions will be put to the test. In a second term, the administration would also likely push to continue torevamp Medicaidwith its efforts to institute work requirements for adult enrollees and provide more flexibility for states to change the contours of the program.

If former Vice President Joe Biden wins and Democrats gain a Senate majority, it would represent the first time the party has controlled the White House and both houses of Congress since 2010 the year the ACA was passed. A top priority will be dealing with the COVID-19 pandemic and the economic fallout. Biden made that a keystone of his campaign, promising to implement policies based on advice from medical and scientific advisers and provide more directives and aid to the states.

But also high on his agenda will beaddressing parts of the ACAthat havent worked as well as its authors hoped. He pledged to add a government-run public option, which would be an alternative to private insurance plans on the marketplaces, and to lower the eligibility age for Medicare to 60.

While Democrats will continue to control the House, the final makeup of the Senate is still to be determined. And even if the Democrats win the Senate, they are not expected to come away with a majority that would allow them to pass legislation without support from at least some GOP senators, unless they change the Senates rules. That could lower expectations of what the Democrats can accomplish andmay lead to some tensionsamong members.

But who controls Washington, D.C., is only part of the elections impact on health policy. Several key health issues are on the ballot both directly and indirectly in many states. Here are a few:

In Colorado, a measure that would have banned abortions after 22 weeks of pregnancy except to save the life of the pregnant person failed, according to The Associated Press. Colorado is one of seven states that dont prohibit abortions at some point in pregnancy. It is also home to one of the few clinics in the nation that perform abortions in the third trimester, often for severe medical complications. The clinic drawspatients from around the nation, so residents of other states would have been affected if the Colorado amendment passed.

In Louisiana, however, voters easily approved an amendment to the state constitution to say that nothing in the document protects the right to, or requires the funding of, abortion. That would make it easier for the state to outlaw abortion if the Supreme Court overturnsRoe v. Wade, which makes state abortion bans unconstitutional.

The fate of the Medicaid program, for people with low incomes, was not on the ballot directly anywhere this election. (Voters approved expansions of the program inMissouriandOklahomaearlier this year.) But the program will be affected not only by who controls the presidency and Congress, but also by who controls the legislatures in states that have not expanded the program under the Affordable Care Act.North Carolinais a key swing state where a change in majority in the legislature could turn the expansion tide.

In six states, voters were deciding the legality of marijuana in one form or another. Montana, Arizona and New Jersey were deciding whether to join the 11 states that allow recreational use of the drug. Mississippi and Nebraska voters were choosing whether to legalize medical marijuana, and South Dakota was the first state to vote on legalizing both recreational and medical pot in the same election.

Magic mushrooms were on two ballots. A measure in Oregon to allow the use of psilocybin-producing mushrooms for medicinal purposes passed, and a District of Columbia proposal to decriminalize the hallucinogenic fungi was leading.

Also approved was a separate ballot question in Oregon to decriminalize possession of small amounts of hard drugs, including heroin, cocaine and methamphetamine, and mandate establishing addiction recovery centers, using some tax proceeds from marijuana sales to establish those centers.

As usual, voters in California faced a lengthy list of health-related ballot measures.

For the second time in two years, the states profitable kidney dialysis industry was challenged at the ballot box. Aunion-sponsored initiativewould have required dialysis companies to employ a doctor at every clinic and submit infection reports to the state. But the industry spent $105 million against the measure. It failed, according to AP.

Voters were also asked to decide, again, whether tofund stem cell researchthrough the California Institute for Regenerative Medicine via Proposition 14. Voters first approved funding for the agency in 2004, and since then, billions have been spent with few cures to show for it. The measure was winning in early returns.

California has been at the forefront of the fight over the so-called gig economy, and this years ballot included aproposal pushed by ride-hailing companies like Uber and Lyftthat would let them continue to treat drivers as independent contractors instead of employees. Under Proposition 22, the companies would not have to provide direct health benefits to drivers but would have to give those who qualify a stipend they could use toward a premium for health insurance purchased through the states individual marketplace, Covered California. The measure was approved.

Finally, voters in the Golden State were asked whether toimpose higher property taxeson commercial property owners with land and property holdings valued at $3 million or more, which could help provide new revenue earmarked for economically struggling cities and counties hit hard by COVID-19, as well as K-12 schools and community colleges. Community clinics, California nurses and Planned Parenthood jumped into the thorny political battle over Proposition 15 taking on powerful business groups eyeing revenue to help rebuild Californias underfunded public health system. The measure was too close to call in early returns.

Democrats in California, who control all statewide elected offices and hold a supermajority in the legislature, had been positioning for a Biden win, and some were already penning ambitious health care legislation for next year. Should Biden win, they said, they plan to crack down on hospital consolidation and end surprise emergency room bills, and some were quietly discussing liberal initiatives such as pursuing a single-payer health care system and expanding Medicaid to cover more unauthorized immigrants.

This story was originally published byKaiser Health Newson November 4 2020. Read the original storyhere.

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How the Election Could Affect Health Care and Drug Policy - Scientific American

A Biden Win and Republican Senate Might Lead to Gridlock on Health Issues – Kaiser Health News

By Stem Cell Medicine

By Julie Rovner November 4, 2020

Former Vice President Joe Biden appeared to be inching toward the 270 electoral votes needed to win the presidency Wednesday afternoon, but at the same time it was becoming clearer that Democrats would not take back the Senate majority they lost in 2014. If that bears out, it could well be a prescription for gridlock on health care.

Without a Democratic majority in the Senate, Biden as president could not likely advance many of his top health agenda items including lowering the eligibility age for Medicare to 60, expanding financial assistance for health insurance under the Affordable Care Act, and creating a public option government health plan.

However, a defeat of President Donald Trump would almost certainly stop his administrations efforts to further erode the effectiveness of the ACA and efforts to turn more of the Medicaid program for those with low incomes back to the states.

Even if Biden wins, this is not the outcome Democrats were hoping for and, to some extent, expecting, based on preelection polling. Andy Slavitt, who ran the Centers for Medicare & Medicaid Services during the Obama administration, noted that frustration in a tweet Wednesday. A large disappointment is that many hoped for a significant repudiation of Trump & his indifference to human life, human suffering, his corruption, and goal of getting rid of the ACA. No matter the final total it will be hard to make that claim, Slavitt said.

Still up in the air is how willing a Republican-led Senate will be to provide further relief to individuals, businesses and states hit hard by the coronavirus epidemic, and whether they will participate in previously bipartisan efforts to curtail surprise out-of-network medical bills and get a handle on prescription drug prices.

To be sure, nothing is final as several key swing states, including Pennsylvania, are still counting thousands of mail-in and early votes. But by midafternoon, Biden was projected by news outlets to have won 253 electoral votes, while Trump had 214. Unofficial returns also showed Biden leading in several states, including Arizona and Nevada, and close to the president in Georgia, where early ballots were still being counted.

Biden was quick to note that nothing is final in brief remarks he made Wednesday afternoon. Im not here to declare that weve won, he said, but I am here to declare that, when the count is finished, we believe that we will be the winners.

Although Democrats poured tens of millions of dollars into races to defeat vulnerable Republican senators this year, most of the incumbents including Sens. Susan Collins (Maine), Steve Daines (Montana), Joni Ernst (Iowa) and Lindsey Graham (South Carolina) won their battles. Several races are still too close to call.

But who controls Washington, D.C., is only part of the elections impact on health policy. Several key health issues were on the ballot both directly and indirectly in many states. Here are a few:

Abortion

In Colorado, a measure that would have banned abortions after 22 weeks of pregnancy except to save the life of the pregnant person failed, according to The Associated Press. Colorado is one of seven states that dont prohibit abortions at some point in pregnancy. It is also home to one of the few clinics in the nation that perform abortions in the third trimester, often for severe medical complications. The clinic draws patients from around the nation, so residents of other states would have been affected if the Colorado amendment had passed.

In Louisiana, however, voters easily approved an amendment to the state constitution to say that nothing in the document protects the right to, or requires the funding of, abortion. That would make it easier for the state to outlaw abortion if the Supreme Court overturns Roe v. Wade, which makes state abortion bans unconstitutional.

Medicaid

The fate of the Medicaid program, for people with low incomes, was not on the ballot directly anywhere this election. (Voters approved expansions of the program in Missouri and Oklahoma earlier this year.) But the program will be affected not only by who controls the presidency and Congress, but also by who controls the legislatures in states that have not expanded the program under the Affordable Care Act. North Carolina is a key swing state where a change in majority in the legislature could have turned the expansion tide, but Republicans held onto their majority.

Drug Policy

In six states, voters were deciding the legality of marijuana in one form or another. Montana, Arizona and New Jersey opted to join the 11 states that allow recreational use of the drug. Mississippi voters agreed to legalize medical marijuana, and South Dakota approved legalizing both recreational and medical pot.

Magic mushrooms were on two ballots. A measure in Oregon to allow the use of psilocybin-producing mushrooms for medicinal purposes passed, as did a District of Columbia proposal to decriminalize the hallucinogenic fungi.

Also approved was a separate ballot question in Oregon to decriminalize possession of small amounts of hard drugs, including heroin, cocaine and methamphetamine, and mandate establishing addiction recovery centers, using some tax proceeds from marijuana sales to establish those centers.

California

As usual, voters in California faced a lengthy list of health-related ballot measures.

For the second time in two years, the states profitable kidney dialysis industry was challenged at the ballot box. A union-sponsored initiative would have required dialysis companies to employ a doctor at every clinic and submit infection reports to the state. But the industry spent $105 million against the measure. It failed, according to unofficial returns posted by the state.

Voters were also asked to decide, again, whether to fund stem cell research through the California Institute for Regenerative Medicine via Proposition 14. Voters first approved funding for the agency in 2004, and since then, billions have been spent with few cures to show for it. The measure passed.

California has been at the forefront of the fight over the so-called gig economy, and this years ballot included a proposal pushed by ride-hailing companies like Uber and Lyft that would let them continue to treat drivers as independent contractors instead of employees. Under Proposition 22, the companies would not have to provide direct health benefits to drivers but would have to give those who qualify a stipend they could use toward a premium for health insurance purchased through the states individual marketplace, Covered California. The measure was approved.

Finally, voters in the Golden State were asked whether to impose higher property taxes on commercial property owners with land and property holdings valued at $3 million or more, which could help provide new revenue earmarked for economically struggling cities and counties hit hard by COVID-19, as well as K-12 schools and community colleges. Community clinics, California nurses and Planned Parenthood jumped into the thorny political battle over Proposition 15 taking on powerful business groups eyeing revenue to help rebuild Californias underfunded public health system. The measure appeared to have failed in a close vote.

Democrats in California, who control all statewide elected offices and hold a supermajority in the legislature, had been positioning for a Biden win, and some were already penning ambitious health care legislation for next year. Should Biden win, they said, they plan to crack down on hospital consolidation and end surprise emergency room bills, and some were quietly discussing liberal initiatives such as pursuing a single-payer health care system and expanding Medicaid to cover more unauthorized immigrants.

JoNel Aleccia, Rachel Bluth, Angela Hart, Matt Volz and Samantha Young contributed to this story.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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A Biden Win and Republican Senate Might Lead to Gridlock on Health Issues - Kaiser Health News

Blockchain offers advanced therapy tracking – Bioprocess InsiderBioProcess – BioProcess Insider

By Gene Therapy Clinics

Hataali blockchain technology used to secure supply chain in the UK could lead to a standardized infrastructure for cell and gene therapy tracking, says ATMPS.

ATMPS, a spin out from Farmatrust and the creator of the Hataali platform, tested the blockchain solution alongside the UKs University Hospitals Birmingham NHS Foundation Trust (UHB).

UHB is home to the Midlands and Wales Advanced Therapy Treatment Centre, which is comprised of partners from the NHS, academia and industry. The center, one of three across the UK, delivers advanced therapies to UK patients.

Image: iStock/ipopba

Hataali was tested alongside the UHBs system, where it was found that the blockchain platform was interoperable, with ATMPS suggesting that its platform had become the first functional blockchain solution for advanced therapeutic products.

The platform is utilized to record scheduling and ordering data for advanced therapy medicinal product (ATMP) treatments within a blockchain system that enables secure and confidential sharing of data between partners.

The solution allows for the activities of manufacturers, clinics, hospitals, and specialist couriers to be shared digitally, as well as being able to provide a regulatory audit trail. Essentially, a digital chain of records is created that allows for communication along each step of the supply chain.

In terms of what benefits this provides over existing platforms and the level of information available to stakeholders, a spokesperson for ATMPS outlined: Its not just an improvement on, say, serialization, as it gives on open access window to access user appropriate data e.g. doctors can better plan appointments, regulators can be secure in the supply chain conditions, and patients and innovators can know exactly where the treatment is, and crucially be alerted if there is a problem.

Now that the trial is complete, ATMPS plans to use this initial rollout as an example to widen the use of the platform. The spokesperson described the partnership with UHB as just the start of the process, with the platform designed to be multi-treatment, multi-site and multi-stakeholder.

This sees ATMPS considering the next stage for the platform being UK-wide utilization. Eventually, however, the company foresees a global structure whereby each new advanced therapy that gets approved in various markets enters a standardized infrastructure.

For Hataali itself, this means that the developers have already created the platform to function across time zones and to include multiple languages.

The patent for the platform is currently still pending, which means that rivals could potentially create an alternative platform based on the same premise.

There are already existing tracking platforms available on the market, though their scope through the supply chain is more limited, such as TrakCels supply chain monitoring technology that is able to track manufacturing processes in real-time for cell and gene therapies.

However, the spokesperson for ATMPS was unconcerned about potential rivals, Our solution is unique in that it is the only vein-to-vein solution out there that uses a blockchain foundation. So, even while its patent pending, I dont think we will see many investors trying to bring forward a competitive solution, they explained.

Furthermore, blockchain is complicated technically and any platform developed with this market in mind is required to be compliant with HIPAA and GDPR regulations, they added.

ATMPS spokesperson observed that those working in blockchain hold the ethos and mindset that collaborating and competing is the best way to push forward their own company, and healthcare more broadly.

They concluded, We are always keen to explore joint opportunities and to work with other solution providers that are operating in this novel specialist area, and we have no problem with anyone working or integrating with us, competitors or not.

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Blockchain offers advanced therapy tracking - Bioprocess InsiderBioProcess - BioProcess Insider

Flexion Therapeutics Reports Third-Quarter 2020 Financial Results and Recent Business Highlights – GlobeNewswire

By Gene Therapy Clinics

November 04, 2020 16:01 ET | Source: Flexion Therapeutics, Inc.

BURLINGTON, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) today reported financial results and recent business highlights for the quarter ended September30, 2020.

Throughout the third quarter, we saw strong execution across all areas of our business, and we were particularly pleased to achieve $23.7million in ZILRETTA net sales, said Michael Clayman, M.D., President and Chief Executive Officer of Flexion Therapeutics. We firmly believe that ZILRETTA can play an increasingly prominent role in helping the millions of patients who confront osteoarthritis knee pain, and our confidence in its long-term potential has only grown. Furthermore, during the quarter we made outstanding progress with our pipeline of innovative product candidates. We advanced FX201, our investigational intra-articular gene therapy product candidate for osteoarthritis, into the second cohort of the Phase 1 safety and tolerability study, and we remain on track to file an IND and potentially initiate clinical trials for FX301, our locally administered peripheral nerve block candidate for control of postoperative pain in 2021.

Third-Quarter Results & Financial Highlights

The Company reported a net loss of $24.6million for the third quarter of 2020, compared to a net loss of $38.2million for the same period of 2019. Net sales of ZILRETTA were $23.7million and $21.8million for the three months ended September 30, 2020 and 2019, respectively.

Cost of sales was $5.1million and $2.9million for the three months ended September 30, 2020 and 2019, respectively. For the three months ended September 30, 2020, cost of sales included $2.0million for the actual cost of units sold and $3.1million of unabsorbed overhead associated with the voluntary, temporary suspension of manufacturing activities at Patheon due to COVID19 impacts on sales of ZILRETTA. For the three months ended September30, 2019, cost of sales was comprised of $1.8million related to the actual cost of units sold, $0.9million related to unabsorbed manufacturing costs and $0.2million of period costs and other adjustments.

Research and development expenses were $10.1million and $20.9million for the three months ended September30, 2020 and 2019, respectively. The decrease in research and development expenses of $10.8million was primarily due to a decrease of $5.9million in development expenses for ZILRETTA due to a reduction in ZILRETTA life cycle management activities, a decrease in $2.9million in portfolio expansion-related expenses as the upfront payment for the purchase of XEN402 from Xenon Pharmaceuticals occurred in the third quarter of 2019, as well as a decrease of $1.4million in salary and other employee-related costs and stock-based compensation expense related to lower headcount.

Selling, general and administrative expenses were $27.3million and $32.1million for the three months ended September30, 2020 and 2019, respectively. Selling expenses were $19.3million and $23.9million for the three months ended September30, 2020 and 2019, respectively. The year-over-year decrease of $4.6million was primarily due to the expense reduction measures taken in response to COVID19; in particular, the elimination of live presence at industry conferences, reductions in in-person physician speaker programs and reductions in select marketing programs and materials, as well as a reduction in travel expenses due to physician office limitations and travel guidelines and restrictions at the state and local level. General and administrative expenses were $8.0million and $8.2million for the three months ended September30, 2020 and 2019, respectively, which represents a decrease of $0.2million.

Flexion expects full-year 2020 total operating expenses (including cost of sales, research and development, and selling, general and administrative) will be in the range of $172million to $182million.

Interest income was $0.1million and $0.7million for the three months ended September30, 2020 and 2019, respectively. Interest expense was $5.1million and $4.7million for the three months ended September30, 2020 and 2019, respectively.

As of September30, 2020, the Company had approximately $185.3million in cash, cash equivalents and marketable securities compared with $136.7million as of December31, 2019. Based on the Companys current operating plan and assuming purchasing activity at physician practices, clinics and certain medical centers or hospitals that administer ZILRETTA continue to return to pre-COVID19 levels, Flexion believes that its current cash, cash equivalents and marketable securities will be sufficient to fund operations into 2022.

Recent News & Business Updates

Indication and Select Important Safety Information for ZILRETTA

Indication:ZILRETTA is indicated as an intra-articular injection for the management of OA pain of the knee.

Limitation of Use: The efficacy and safety of repeat administration of ZILRETTA have not been demonstrated.

Contraindication:ZILRETTA is contraindicated in patients who are hypersensitive to triamcinolone acetonide, corticosteroids or any components of the product.

Warnings and Precautions:

Adverse Reactions:The most commonly reported adverse reactions (incidence 1%) in clinical studies included sinusitis, cough, and contusions.

Please seeZilrettaLabel.comfor full Prescribing Information.

About ZILRETTA (triamcinolone acetonide extended-release injectable suspension) On October6, 2017, ZILRETTA was approved by the U.S. FDA as the first and only extended-release intra-articular therapy for patients confronting OA-related knee pain. ZILRETTA employs proprietary microsphere technology combining triamcinolone acetonidea commonly administered, short-acting corticosteroidwith a poly lactic-co-glycolic acid (PLGA) matrix to provide extended pain relief. The pivotal Phase 3 trial on which the approval of ZILRETTA was based showed that ZILRETTA significantly reduced OA knee pain for 12 weeks, with some people experiencing pain relief through Week 16. Learn more at http://www.zilretta.com.

About FX201 (humantakinogene hadenovec) FX201 is a novel, intra-articular gene therapy product candidate that utilizes a helper-dependent adenovirus [HDAd] vector based on human serotype 5 (Ad5) that is designed to transfer a gene to cells in the joint to produce an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), under the control of an inflammation-sensitive promoter. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of OA. By persistently suppressing inflammation, Flexion believes FX201 holds the potential to provide long-term pain relief and functional improvement, and to modify the disease.

About FX301 (funapide formulated in a proprietary thermosensitive hydrogel) FX301 is a locally administered NaV1.7 inhibitor product candidate, known as funapide, formulated for extended release in a thermosensitive hydrogel. The initial development of FX301 is intended to support administration as a peripheral nerve block for control of post-operative pain. Flexion believes FX301 has the potential to provide effective and durable pain relief while preserving motor function and anticipates initiating clinical trials in 2021.

About Osteoarthritis (OA) of the Knee OA, also known as degenerative joint disease, affects more than 30million Americans and accounts for more than $185billion in annual expenditures. In 2017, approximately 15million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About Flexion Therapeutics Flexion Therapeutics (Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The Company's core values are focus, ingenuity, tenacity, transparency and fun. Please visitflexiontherapeutics.com.

Forward-Looking StatementsThis release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts are forward looking statements, including, but not limited to, statements relating to the future of Flexion; expectations related to Flexions expenses for the year ended December31, 2020, and cash runway; timing and plans with respect to clinical trials; expected impacts from COVID-19 and the timing and duration of such impacts; the long-term potential of ZILRETTA; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of ZILRETTA and our product candidates. These forward-looking statements are based on managements expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, the fact that the impacts and expected duration of the COVID19 pandemic are uncertain and rapidly changing; risk that we may not achieve expense expectations for 2020; the potential for unexpected expenditures or cash requirements; the risk that we may not be able to successfully maintain an effective sales force to commercialize ZILRETTA; competition from alternative therapies; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to ZILRETTA; the risk that ZILRETTA may not be successfully commercialized or adopted; risks regarding our ability to obtain adequate reimbursement from payers for ZILRETTA; risks related to the manufacture and distribution of ZILRETTA, including our reliance on sole sources of supply and distribution; risks related to clinical trials, including potential delays, safety issues, or negative results; risks related to key employees, markets, economic conditions, health care reform, prices, and reimbursement rates; and other risks and uncertainties described in our filings with the Securities and Exchange Commission (SEC), including under the heading Risk Factors in our Quarterly Report on Form10-Q for the quarter ended June30, 2020, filed with the SEC on August5, 2020, and subsequent filings with the SEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contact:

Scott Young Vice President, Corporate Communications & Investor Relations T: 781-305-7194 syoung@flexiontherapeutics.com

Julie Downs Associate Director, Corporate Communications & Investor Relations T: 781-305-7137 jdowns@flexiontherapeutics.com

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Flexion Therapeutics Reports Third-Quarter 2020 Financial Results and Recent Business Highlights - GlobeNewswire

Lynparza approved in the EU as 1st-line maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer | Small Molecules | News…

By Gene Therapy Clinics

Details Category: Small Molecules Published on Thursday, 05 November 2020 11:36 Hits: 85

Patients treated with Lynparza and bevacizumab lived without disease progression for a median of 37.2 months vs. 17.7 months with bevacizumab alone

One in two women with advanced ovarian cancer has an HRD-positive tumour

LONDON, UK I November 5, 2020 I AstraZeneca and MSDs Lynparza (olaparib) has been approved in the European Union (EU) for the 1st-line maintenance treatment with bevacizumab of patients with homologous recombination deficient (HRD)-positive advanced ovarian cancer.

Ovarian cancer is the fifth most common cause of cancer death in the EU and the five-year survival rate is approximately 45%, due partly because women are often diagnosed with advanced disease (Stage III or IV).1-3

The approval by the European Commission was based on a biomarker subgroup analysis of the PAOLA-1 Phase III trial which showed Lynparza, in combination with bevacizumab maintenance treatment, demonstrated a substantial progression-free survival (PFS) improvement versus bevacizumab alone for patients with HRD-positive advanced ovarian cancer. It follows the recommendation for approval by the Committee for Medicinal Products for Human Use of the European Medicines Agency in September 2020.

Isabelle Ray-Coquard, principal investigator of the PAOLA-1 Phase III trial and medical oncologist, Centre Lon Brard and President of the GINECO group, Paris, France, said: For women with advanced ovarian cancer, the goal of 1st-line treatment is to delay disease progression for as long as possible with the intent of achieving long-term remission. Unfortunately, once a patients cancer recurs, it historically has been incurable. Lynparza together with bevacizumab has demonstrated an impressive median progression-free survival benefit of more than three years and is poised to become the standard of care for eligible patients with HRD-positive tumours in the EU.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: Half of all newly diagnosed patients with advanced ovarian cancer have HRD-positive tumours. Women treated with Lynparza in combination with bevacizumab in the PAOLA-1 Phase III trial lived progression free for a median of more than three years, showing that HRD testing should be an essential component of clinical diagnosis. HRD status can help physicians select a personalised 1st-line treatment regimen for patients to substantially delay relapse in this devastating disease.

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: Biomarker testing has rapidly enhanced our understanding of how PARP inhibition can help target this disease. The EU approval reinforces that HRD-positive tumours represent a distinct subset of advanced ovarian cancer and HRD testing is critical for women in this setting.

The PAOLA-1 Phase III trial showed thatLynparza,in combination with bevacizumab maintenance treatment, reduced the risk of disease progression or death by 67% (based on a hazard ratio of 0.33; 95% confidence interval 0.25-0.45). The addition ofLynparzaimproved PFS to a median of 37.2 months versus 17.7 with bevacizumab alone in patients with HRD-positive advanced ovarian cancer. The data from the PAOLA-1 trial was published inThe New England Journal of Medicinein 2019.

Further results recently presented at the European Society for Medical Oncology Virtual Congress 2020 showed a statistically significant improvement in the key secondary endpoint of the time to second disease progression (PFS2). Lynparza with bevacizumab provided benefit beyond first disease progression, improving PFS2 to a median of 50.3 months versus 35.3 with bevacizumab alone.

The full EU indication is for Lynparza in combination with bevacizumab for the maintenance treatment of adult patients with advanced (FIGO Stages III and IV) high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of 1st-line platinum-based chemotherapy in combination with bevacizumab and whose cancer is associated with HRD positive status defined by either a breast cancer susceptibility gene 1/2 (BRCA1/2) mutation and/or genomic instability.

Lynparzain combination with bevacizumab isapproved in the USand in several other countries as a 1st-line maintenance treatment for patients with HRD-positive advanced ovarian cancer and is currently under regulatory review in other countries around the world.

Financial considerations

Following this approval for Lynparza in the EU, AstraZeneca will receive a regulatory milestone payment from MSD of $25m, anticipated to be booked as collaboration revenue during the fourth quarter of 2020.

Ovarian cancer

In 2018, there were nearly 68,000 new cases of ovarian cancer diagnosed in the EU and around 45,000 deaths.3Approximately 50% of ovarian cancers are HRD-positive including BRCA1/2 mutation.4,5Approximately 15% of ovarian cancers have a BRCA1/2 mutation.6 The primary aim of 1st-line treatment is to delay disease progression for as long as possible with the intent to achieve long-term remission.7-9

Homologous recombination deficiency

HRD, which defines a subgroup of ovarian cancer, encompasses a wide range of genetic abnormalities, including BRCA mutations and beyond. As with BRCA gene mutations, HRD interferes with normal cell DNA repair mechanisms and confers sensitivity to PARP inhibitors including Lynparza.10

PAOLA-1

PAOLA-1 is a double-blinded Phase III trial testing the efficacy and safety ofLynparzaadded to standard-of-care bevacizumab versus bevacizumab alone, as a 1st-line maintenance treatment for newly diagnosed advanced FIGO Stage III-IV high-grade serous or endometroid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to 1st-line treatment with platinum-based chemotherapy and bevacizumab.AstraZeneca and MSD announced in August 2019 that the trial met its primary endpoint of PFS in the overall trial population.

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair (HRR), such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US as a 1st-line maintenance treatment with bevacizumab for patients with HRD-positive advanced ovarian cancer (BRCAm and/or genomic instability). Lynparza is approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is also approved in the US, the EU and several other countries for the treatment of germline BRCAm metastatic pancreatic cancer. Lynparza is approved in the US for homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (BRCAm and other HRR gene mutations). Regulatory reviews are underway in several countries for ovarian, breast, pancreatic and prostate cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 30,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca's industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the worlds first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio ofnew medicines that has the potential to transform patients lives and the Companys future. With seven new medicines launched between 2014 and 2020, and a broad pipelineof small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers.

By harnessing the power of four scientific platforms Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.

AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.

References

1. EuroHealth. (2018). Ovarian Cancer: The Silent Killer. Available at: https://eurohealth.ie/policy-brief-women-and-ovarian-cancer-in-the-eu-2018/ [Accessed October 2020].

2. ECIS. (2020).Estimates of cancer incidence and mortality in 2020, for all cancer sites. Available here [Accessed October 2020].

3. The World Health Organization. IARC. Globocan. (2018). Available at: http://gco.iarc.fr/ [Accessed October 2020].

4. Moschetta et al. (2016). BRCA somatic mutations and epigenetic BRCA modifications in serous ovarian cancer. Annals of Oncology, 27(8), pp.1449-1455.

5. Bonadio et al. (2018). Homologous recombination deficiency in ovarian cancer: a review of its epidemiology and management. Clinics, 73(Suppl 1): e450s.

6. Ramus. (2009). The Contribution of BRCA1 and BRCA2 to Ovarian Cancer. Molecular Oncology, 3(2), pp.138150.

7. Raja et al. (2012). Optimal first-line treatment in ovarian cancer. Annals on Oncology. 23 Suppl 10, x118-127.

8. NHS Choices, Ovarian Cancer Available at: https://www.nhs.uk/conditions/ovarian-cancer/treatment/ [Accessed October 2020].

9. Ledermann et al. (2013). Newly diagnosed and relapsed epithelial ovarian carcinoma: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology, 24, pp.vi24-vi32.

10. Moore, K. (2018). Maintenance Olaparib in Patients with Newly Diagnosed Advanced Ovarian Cancer. New England Journal of Medicine, 379(26), pp.2495-2505.

SOURCE: AstraZeneca

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Predictive Biomarkers Market Growth Sales Revenue Analysis 2020-2025 – The Think Curiouser

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